Held May 1, 2026
The voices of the DBAS community, on the record
On 1 May 2026, the Styrke Foundation hosted an Externally Led Patient-Focused Drug Development meeting for Diamond-Blackfan Anemia Syndrome.
Patients, caregivers, clinicians and staff from the U.S. Food and Drug Administration met to discuss what living with DBAS asks of families and what better treatment would mean.
Watch the Meeting in full
The complete recording includes:
- Patient and caregiver testimony,
- Introductory remarks from Dr. Kavita Natrajan from the US Food and Drug Administration (FDA),
- A clinical overview of DBAS from Dr Josu de la Fuente,
- Discussion of current treatments led by Dr. Christine Duncan,
- The open discussions that followed each topic.
What happened on the day
The meeting was structured around two topics. The first focused on health effects and daily impact. The second focused on current approaches to treatment.
Patient and caregiver panelists opened each topic, followed by a large-group discussion with attendees joining from around the world.
The day was supported by a Patient and Caregiver Survey, which gathered responses from 82 patients and caregivers across 9 countries.
Together, the panel testimony and the survey data give the FDA a wider evidence base for understanding what the DBAS community is asking for.
Next Steps
The Voice of the Patient report
The Styrke Foundation is now compiling the Voice of the Patient report based on the testimony shared during the meeting and the supporting survey. Once finalised, the report will be submitted to the FDA and made publicly available, where it can inform regulatory decisions, clinical trial design, and the broader development of treatments for DBAS.
We will keep advocating alongside the patient community as gene therapy programmes and other treatments progress through development.
Patient and Caregiver Survey
As we prepare the VOP over the next few months, we will leave the survey open and ask people to continue take the survey and comment.
Your voice will be accounted for in our report.
With Thanks
The meeting would not have been possible without the people who agreed to speak on the record about a private and difficult subject. We are grateful to:
Patient & caregiver panellists
Who shared their experiences openly
Dr Kavita Natrajan
For the introductory remarks
Dr. Josu de la Fuente
For the clinical overview
Dr. Christine Duncan
For the discussion of current treatments
FDA staff
Who attended and engaged with the community
Survey respondents
80+ patients and caregivers across 9 countries
The wider DBAS community
Whose advocacy made this meeting possible
Why These Meetings Matter
Direct Patient Input in Drug Development Process
Externally-led Patient-Focused Drug Development meetings provide FDA and key stakeholders—including medical product developers, healthcare providers, and federal partners—with crucial opportunities to hear directly from patients, families, caregivers, and patient advocates.
Understanding Patient Priorities and Daily Disease Impact
These meetings focus on gathering insights about the symptoms that matter most to patients, how the disease affects their daily lives, and their real-world experiences with currently available treatments.
Informing FDA Regulatory Decisions and Oversight
Patient input from meetings and meeting summary reports can support FDA staff:
- In conducting benefit-risk assessments for products under review, by informing the therapeutic context
- Advising drug sponsors on their development programs
Patient input can support drug development more broadly:
- Identify areas of unmet need in the patient population
- Identify or develop tools that assess benefit of potential therapies
- Raise awareness and increase engagement within the patient community
Resources
Links to Past Meeting Materials and Reports
Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience Webpage All FDA-led PFDD meeting materials (agenda, slides, recordings, etc.) are available on this page.
Alphabetical listing of condition-specific meeting reports and other information related to patients’ experience.
These meetings include FDA-led Patient-Focused Drug Development (PFDD) meetings, Externally-led PFDD meetings, and Patient Listening Sessions.